多发性硬化
视神经脊髓炎
医学
再髓鞘化
临床试验
光谱紊乱
髓鞘少突胶质细胞糖蛋白
疾病
神经影像学
神经科学
重症监护医学
病理
免疫学
髓鞘
心理学
精神科
中枢神经系统
内科学
实验性自身免疫性脑脊髓炎
作者
Giulia Fadda,Thaís Armangué,Yael Hacohen,Tanuja Chitnis,Brenda Banwell
标识
DOI:10.1016/s1474-4422(20)30432-4
摘要
The field of acquired CNS neuroimmune demyelination in children is transforming. Progress in assay development, refinement of diagnostic criteria, increased biological insights provided by advanced neuroimaging techniques, and high-level evidence for the therapeutic efficacy of biological agents are redefining diagnosis and care. Three distinct neuroimmune conditions—multiple sclerosis, myelin-oligodendrocyte glycoprotein antibody-associated disease (MOGAD), and aquaporin-4 antibody-associated neuromyelitis optica spectrum disorder (AQP4-NMOSD)—can now be distinguished, with evidence from humans and animal models supporting distinct pathobiological disease mechanisms. The development of highly effective therapies for adult-onset multiple sclerosis and AQP4-NMOSD that suppress relapse rate by more than 90% has motivated advocacy for trials in children. However, doing clinical trials is challenging because of the rarity of these conditions in the paediatric age group, necessitating new approaches to trial design, including age-based trajectory modelling based on phase 3 studies in adults. Despite these limitations, the future for children and adolescents living with multiple sclerosis, MOGAD, or AQP4-NMOSD is far brighter than in years past, and will be brighter still if successful therapies to promote remyelination, enhance neuroprotection, and remediate cognitive deficits can be further accelerated.
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